On May 12, 2022 X4 Pharmaceuticals, (Nasdaq: XFOR), a leader in the discovery and development of novel oral CXCR4-targeted small molecule therapeutics to benefit people with rare immune system disorders, reported financial results for the first quarter ended March 31, 2022 (Press release, X4 Pharmaceuticals, MAY 12, 2022, View Source [SID1234614391]).

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"As we quickly approach top-line results from our 4WHIM pivotal Phase 3 trial for WHIM syndrome by the end of this year, the X4 team continues to identify and expand the breadth of individuals diagnosed with this rare disease. Initiatives aimed at supporting diagnosis and driving disease awareness, such as our PATH4WARD program offering free genetic sequencing to patients, and publishing peer-reviewed research to help define novel pathogenic genetic variants, are adding to the population of those known to be affected by WHIM and chronic neutropenia," said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. "Specifically, to support those affected by chronic neutropenia, we are expanding the availability of genetic testing through PATH4WARD and evaluating patients with congenital, idiopathic, and cyclic chronic neutropenia in our ongoing Phase 1b study. We look forward to providing both clinical data and regulatory updates over the coming months on this important initiative."

Recent Highlights

Presented posters at the CIS 2022 Annual Meeting that detailed the PATH4WARD genetic testing program to aid in the diagnosis of WHIM syndrome and chronic neutropenia, and the characterization of a novel pathogenic CXCR4 variant (S341Y).
Presented preclinical data on mavorixafor’s ability to significantly enhance the tumor-cell killing activity of the leading commercial and clinical stage Bruton Tyrosine Kinase Inhibitors (BTKi) including ibrutinib, zanubrutinib, pirtobrutinib (LOXO-305) and nemtabrutinib (ARQ-531), at the 2022 AACR (Free AACR Whitepaper) Annual Meeting.
Completed enrollment in the ongoing Phase 1b study of mavorixafor in combination with ibrutinib in patients with Waldenström’s Macroglobulinemia (WM) whose tumors express mutations in MYD88 and CXCR4. A total of 16 patients were enrolled and dose escalation to the 600 mg dose of mavorixafor has been completed. Results from this study are expected to be reported during the second half of 2022.

First Quarter 2022 Financial Results

Cash, Cash Equivalents & Restricted Cash: X4 had $67.7 million in cash, cash equivalents, and restricted cash as of March 31, 2022. The company expects that its cash and cash equivalents will fund company operations into the fourth quarter of 2022.
Research and Development (R&D) Expenses were $14.1 million for the first quarter of 2022 as compared to $12.1 million for the comparable period in 2021. R&D expenses include $0.7 million and $0.6 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Selling, General and Administrative Expenses (SG&A) were $7.7 million for the first quarter of 2022 as compared to $5.8 million for the comparable period in 2021. SG&A expenses include $0.8 million and $0.7 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Net Loss: X4 reported a net loss of $22.0 million for the first quarter of 2022, as compared to $18.7 million for the comparable period in 2021. Net losses include $1.5 million and $1.3 million of certain non-cash expenses for the first quarter of 2022 and 2021, respectively.
Conference Call and Webcast
X4 will host a conference call and webcast today at 8:30 am EDT to discuss financial results and business highlights. The conference call can be accessed by dialing (866) 721-7655 from the United States or (409) 216-0009 internationally, followed by the conference ID: 4382059. The live webcast can be accessed on the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay of the conference call will be available on the website.